Monitoring Of Patient-reported Outcomes Research Articles

PRO-WAVE1: Monitoring patient reported outcomes (PROs) in prostate cancer through Wave Health, a novel ePRO health platform.

e23190 Background: It is well established that the monitoring and evaluation of PROs in cancer patients has a positive impact in both quality of life and overall survival [Basch E., JAMA 2017]. In parallel, the widespread use of electronic devices such as smart phones in the general population have increased significantly over recent years [Statista, 2023], representing a unique opportunity to operationalize ePROs. However, there is uncertainty about the feasibility and applicability of these technologies in certain patient population such as prostate cancer (PC) patients. PRO-WAVE1 aims to determine the acceptability and engagement of the Wave Health patient app and healthcare provider portal, enhanced for PC patients. Specific endpoints include measuring longitudinal use of varying ePROs to impact care management, patient health literacy, and ability to identify and manage emerging risks. Methods: Patients across eight sites in Spain engaged with the Wave Health App over a 13-week period. Each week, patients completed a validated weekly symptom review (WSR) assessing 16 Prostate cancer-specific symptoms, including severity and/or frequency of each; as well as a health-related QoL (FACT-P) and health literacy survey (CHLT-30-Dkspa). A report summary of the patients’ WSR was generated for care team view in the Wave Health Connect platform – specifically to conduct weekly check ins on patients, including verifying patient completion; evaluating report content; indicating plans for follow up per patient; and rating their perceived value of the platform. Results: 98 patients were enrolled in the study – of which 82 actively participated in the study (84% acceptance). The compliance rate for the ePRO questionnaires was 93.3%, while during their participation, patients used the app for an average of 12.2 weeks, and approximately two-thirds (67.4%) used the app for all 13 weeks. Patient satisfaction with the platform was 93%, with 100% of respondents feeling the information provided in the app was relevant to their personal experience. Correspondingly, almost all HCPs (98%) rated the platform as useful – with the majority willing to recommend it to their peers and future patients; 90% of HCPs positively perceived the platform’s impact on the quality of communication with patients. Conclusions: Study results exceeded expectations for patient acceptability and engagement with the app; as well as perceived usefulness of the portal among HCPs – suggesting the platform as an impactful tool for patients working with HCPs to manage treatment journeys. A larger study using a comparator arm will help further evaluate the impact on costs and clinical outcomes.

A randomized study comparing electronic patient-reported outcome (ePRO) monitoring with routine follow-up during trastuzumab deruxtecan treatment in patients with metastatic breast cancer (PRO-DUCE study).

1504 Background: Trastuzumab deruxtecan (T-DXd) is a highly effective agent for HER2-positive metastatic breast cancer (MBC), but is associated with adverse events such as nausea, fatigue, and interstitial lung disease. Based on recent studies showing that symptom monitoring and alert notifications via ePROs could improve patient quality of life (QoL) and prognosis, we hypothesized that employing this method may diminish the treatment burden for patients receiving T-DXd. Methods: In this multicenter, randomized controlled exploratory study (jRCTs031200387), we randomized patients with HER2-positive MBC eligible for T-DXd to either the ePRO monitoring (ePROm) group or the usual care (UC) group. ePROm involved weekly symptom and daily body temperature/SpO2 reports via a smartphone or computer at home. If any symptoms exceeded the predetermined thresholds, an email alert was sent to the medical staff, the ePRO was evaluated, and, if necessary, a phone consultation was provided. The primary endpoint was the change in the Global QoL score of EORTC QLQ-C30 from baseline at week 24. Secondary endpoints included score changes in the functional and symptom scales of EORTC QLQ-C30, and the time to deterioration of Global QoL (defined as a 10-point decrease from baseline). For a score difference between groups of 10 points and a standard deviation of 24, the required number of patients was 55 in each group, with a two-sided alpha error of 10% and a power of 87%. We analyzed the primary endpoint using a mixed-effects model for repeated measures. Results: Between March 2021 and January 2023, 111 patients were enrolled at 38 hospitals in Japan, with 56 assigned to ePROm and 55 to UC; the full-analysis set for QoL assessment included 54 and 52 patients, respectively. During the 24-week period, there were 1223 ePRO reports (95% compliance rate) in the ePROm group, including 427 alert notifications (7.9 per patient) to healthcare providers. Overall compliance with the questionnaire for QoL was 97%. Mean changes from baseline scores in Global QoL at week 24 showed that ePROm was significantly better than UC (mean difference 8.0 [90% CI 0.2, 15.8]; p=0.091). Role, cognitive, and social functioning were better in ePROm, with mean differences of 10.0 (95% CI 1.1, 18.9; p=0.028), 6.3 (1.1, 11.5; p=0.017), and 10.9 (3.9, 18.0; p=0.003), respectively. There was no difference in nausea/vomiting (0.5 [- 6.2, 7.1]; p=0.889), while fatigue was significantly better in ePROm (-8.4 [-16.1, -0.6]; p=0.034). Median time to first deterioration in Global QoL score was 3.9 months (95% CI 2.5, 13.9) in ePROm and 3.0 months (95% CI 1.6, 6.7) in UC (hazard ratio 0.73 [95% CI 0.45, 1.17]; p=0.159). Conclusions: ePROm systems, especially for symptom and SpO2 tracking in HER2-positive MBC patients treated with T-DXd, are promising to enhance patient QoL. Clinical trial information: jRCTs031200387 .

A randomized trial of the impact of symptom monitoring using an electronic patient-reported outcome app on health-related quality of life in postmenopausal breast cancer patients receiving adjuvant endocrine therapy.

Electronic patient-reported outcomes (ePRO) monitoring is a useful communication tool for cancer patients and healthcare providers. In this study, we examined the impact of symptom monitoring using an ePRO app on quality of life (QoL) in postmenopausal breast cancer patients receiving adjuvant endocrine therapy. The free app "Welby My Carte ONC" was used in the study. Patients with breast cancer starting adjuvant endocrine therapy were randomly assigned in a 1:1 ratio to ePRO monitoring (ONC) and control groups. The ONC group reported five symptoms extracted from the Patient-Reported Outcome-Common Terminology Criteria for Adverse Events (PRO-CTCAE) (insomnia, joint pain, headache, anxiety, and hot flashes) weekly for 3months through the app. Reported symptoms were shared with medical personnel. When serious symptoms were reported, these personnel ascertained the patient's health status and provided advice over the phone. The primary endpoint was QoL measured by the Functional Assessment of Cancer Therapy-Breast (FACT-B) at 3months from enrollment. Differences between groups were tested using analysis of covariance. The study included 125 subjects with mean age of 64years in the ONC group (n = 61) and 63years in the control group (n = 64). In the ONC group, the response rate to PRO-CTCAE was about 70% or higher until week 10. The item missing rate was 0. The ONC group reported more symptoms related to joint pain and insomnia. The difference in FACT-B total score between the groups was -1.55 (95% confidence interval: -5.91, 2.81), indicating no significant difference. Symptom monitoring using ePRO early after initiation of adjuvant endocrine therapy after surgery did not improve QoL of breast cancer patients.

Association of Remote Patient-Reported Outcomes and Step Counts With Hospitalization or Death Among Patients With Advanced Cancer Undergoing Chemotherapy: Secondary Analysis of the PROStep Randomized Trial.

Patients with advanced cancer undergoing chemotherapy experience significant symptoms and declines in functional status, which are associated with poor outcomes. Remote monitoring of patient-reported outcomes (PROs; symptoms) and step counts (functional status) may proactively identify patients at risk of hospitalization or death. The aim of this study is to evaluate the association of (1) longitudinal PROs with step counts and (2) PROs and step counts with hospitalization or death. The PROStep randomized trial enrolled 108 patients with advanced gastrointestinal or lung cancers undergoing cytotoxic chemotherapy at a large academic cancer center. Patients were randomized to weekly text-based monitoring of 8 PROs plus continuous step count monitoring via Fitbit (Google) versus usual care. This preplanned secondary analysis included 57 of 75 patients randomized to the intervention who had PRO and step count data. We analyzed the associations between PROs and mean daily step counts and the associations of PROs and step counts with the composite outcome of hospitalization or death using bootstrapped generalized linear models to account for longitudinal data. Among 57 patients, the mean age was 57 (SD 10.9) years, 24 (42%) were female, 43 (75%) had advanced gastrointestinal cancer, 14 (25%) had advanced lung cancer, and 25 (44%) were hospitalized or died during follow-up. A 1-point weekly increase (on a 32-point scale) in aggregate PRO score was associated with 247 fewer mean daily steps (95% CI -277 to -213; P<.001). PROs most strongly associated with step count decline were patient-reported activity (daily step change -892), nausea score (-677), and constipation score (524). A 1-point weekly increase in aggregate PRO score was associated with 20% greater odds of hospitalization or death (adjusted odds ratio [aOR] 1.2, 95% CI 1.1-1.4; P=.01). PROs most strongly associated with hospitalization or death were pain (aOR 3.2, 95% CI 1.6-6.5; P<.001), decreased activity (aOR 3.2, 95% CI 1.4-7.1; P=.01), dyspnea (aOR 2.6, 95% CI 1.2-5.5; P=.02), and sadness (aOR 2.1, 95% CI 1.1-4.3; P=.03). A decrease in 1000 steps was associated with 16% greater odds of hospitalization or death (aOR 1.2, 95% CI 1.0-1.3; P=.03). Compared with baseline, mean daily step count decreased 7% (n=274 steps), 9% (n=351 steps), and 16% (n=667 steps) in the 3, 2, and 1 weeks before hospitalization or death, respectively. In this secondary analysis of a randomized trial among patients with advanced cancer, higher symptom burden and decreased step count were independently associated with and predictably worsened close to hospitalization or death. Future interventions should leverage longitudinal PRO and step count data to target interventions toward patients at risk for poor outcomes. ClinicalTrials.gov NCT04616768; https://clinicaltrials.gov/study/NCT04616768. RR2-10.1136/bmjopen-2021-054675.

Digital Remote Monitoring Using an mHealth Solution for Survivors of Cancer: Protocol for a Pilot Observational Study.

Healthy lifestyle interventions have a positive impact on multiple disease trajectories, including cancer-related outcomes. Specifically, appropriate habitual physical activity, adequate sleep, and a regular wholesome diet are of paramount importance for the wellness and supportive care of survivors of cancer. Mobile health (mHealth) apps have the potential to support novel tailored lifestyle interventions. This observational pilot study aims to assess the feasibility of mHealth multidimensional longitudinal monitoring in survivors of cancer. The primary objective is to test the compliance (user engagement) with the monitoring solution. Secondary objectives include recording clinically relevant subjective and objective measures collected through the digital solution. This is a monocentric pilot study taking place in Bangor, Wales, United Kingdom. We plan to enroll up to 100 adult survivors of cancer not receiving toxic anticancer treatment, who will provide self-reported behavioral data recorded via a dedicated app and validated questionnaires and objective data automatically collected by a paired smartwatch over 16 weeks. The participants will continue with their normal routine surveillance care for their cancer. The primary end point is feasibility (eg, mHealth monitoring acceptability). Composite secondary end points include clinically relevant patient-reported outcome measures (eg, the Edmonton Symptom Assessment System score) and objective physiological measures (eg, step counts). This trial received a favorable ethical review in May 2023 (Integrated Research Application System 301068). This study is part of an array of pilots within a European Union funded project, entitled "GATEKEEPER," conducted at different sites across Europe and covering various chronic diseases. Study accrual is anticipated to commence in January 2024 and continue until June 2024. It is hypothesized that mHealth monitoring will be feasible in survivors of cancer; specifically, at least 50% (50/100) of the participants will engage with the app at least once a week in 8 of the 16 study weeks. In a population with potentially complex clinical needs, this pilot study will test the feasibility of multidimensional remote monitoring of patient-reported outcomes and physiological parameters. Satisfactory compliance with the use of the app and smartwatch, whether confirmed or infirmed through this study, will be propaedeutic to the development of innovative mHealth interventions in survivors of cancer. PRR1-10.2196/52957.

Remote Between Visit Monitoring in Inflammatory Bowel Disease Care: A Qualitative Study of CAPTURE-IBD Participants and Care Team Members.

We recently showed that CAPTURE-inflammatory bowel disease (IBD)-a care coordination intervention comprised of routine remote monitoring of patient-reported outcomes (PRO) and a care coordinator-triggered care pathway-was more effective at reducing symptom burden for patients with IBD compared to usual care. We aimed to understand how patients and care team providers experienced the intervention and evaluate purported mechanisms of action to plan for future implementation. In this study, 205 patients were randomized to CAPTURE-IBD (n = 100) or usual care(n = 105). We conducted semi-structured interviews with 16 of the 100 participants in the CAPTURE-IBD arm and 5 care team providers to achieve thematic saturation. We used qualitative rapid analysis to generate a broad understanding of experiences, perceived impact, the coordinator role, and suggested improvements. Findings highlight that the intervention was acceptable and user-friendly, despite concerns regarding increased nursing workload. Both participants and care team providers perceived the intervention as valuable in supporting symptom monitoring, psychosocial care, and between-visit action plans to improve IBD care and health outcomes. However, few participants leveraged the care coordinator as intended. Finally, participants reported that the intervention could be better tailored to capture day-to-day symptom changes and to meet the needs of patients with specific comorbid conditions (eg, ostomies). Remote PRO monitoring is acceptable and may be valuable in improving care management, promoting tight control, and supporting whole health in IBD. Future efforts should focus on testing and implementing refined versions of CAPTURE-IBD tailored to different clinical settings.

Statistical analysis plan for the PRO B study: open-label, superiority randomised controlled trial of alarm-based patient-reported outcome monitoring in patients with metastatic breast cancer

BackgroundWith an increasing collection of patient-reported outcomes (PROs) to measure health-related quality of life (HRQoL) in oncological patients, there is still a lack of standardised strategies on how to interpret and use these data in patient care. Prior research has shown support for the use of digital PRO monitoring together with alarm systems to notify clinicians when the PRO values are deteriorating. This system has demonstrated advantages in improving HRQoL and increasing survival rates among oncology patients. Hence, we designed the PRO B study, a superiority multi-centre randomised controlled trial, to investigate the effects of alarm-based monitoring in metastatic breast cancer patients in Germany. The study protocol for the PRO B study was published in September 2021, and this manuscript describes a formal statistical analysis plan (SAP) for the PRO B study to improve the transparency and quality of this trial.Methods and designThe trial aimed to recruit 1000 patients with metastatic breast cancer. However, as of the completion of recruitment on June 15, 2023, we have successfully enrolled 924 patients from 52 breast cancer centres. Patients were 1:1 stratified randomised to the intervention and control groups. App-based PRO questionnaires are sent weekly to the intervention group and every 3 months to the control group. Only patients in the intervention group trigger an alarm if their PRO scores deteriorate, and they are subsequently contacted by the local care team within 48 h. The primary outcome is the fatigue score at 6 months, and secondary outcomes are other HRQoL and overall survival. Evaluation of the superiority of the intervention will be done using a linear mixed model with random intercepts for study centres.ConclusionThis detailed SAP defines the main components of the statistical analysis for the PRO B study to assist the statistician and prevent bias in selecting analysis and reporting findings. Version 1 of the SAP was finalised on January 18, 2024.Trial registrationDRKS (German Clinical Trials Register) DRKS00024015. Registered on February 15, 2021.

PRO-WAVE1: Monitoring patient reported outcomes (PROs) in prostate cancer through Wave Health, a novel ePRO health platform.

114 Background: It is well established that the monitoring and evaluation of PROs in cancer patients has a positive impact in both quality of life and overall survival.1 In parallel, the widespread use of electronic devices such as smart phones in the general population have increased significantly over recent years,2 representing a unique opportunity to operationalize ePROs. However, there is uncertainty about the feasibility and applicability of these technologies in certain patient population such as prostate cancer (PC) patients. PRO-WAVE1 aims to determine the acceptability and engagement of the Wave Health patient app and healthcare provider portal, enhanced for PC patients. Specific endpoints include measuring longitudinal use of varying ePROs to impact care management, patient health literacy, and ability to identify and manage emerging risks. Methods: The study is underway across eight sites in Spain. Patient ePRO engagement is being evaluated through the administration of 19 ePRO questionnaires over the course of 13 weeks, including: health-related QoL (FACT-P); health literacy (CHLT-30-Dkspa); and weekly reviews of symptoms (WSR). Compliance rates per patient and per cohort will be reported at assessment time-points and for the entire study period. Secondary objectives evaluated include engagement with Wave Health App; changes in HRQoL; changes in health literacy; and the perceived value of the platform by patients and HCPs. Results: 98 patients were enrolled in the study. Patient cohort representation included: 44.9% mHSPC, 36.7% mCRPC, and 18.3% nmPC. Of the 98 patients, 84 successfully became active in the study and participated (85.7% acceptance). As of September 20, 2023, among the completed patients (N=46), the compliance rate for the ePRO questionnaires was 93.7%. During their participation, these patients used the app for an average of 12.2 weeks, and approximately two-thirds (67.4%) used the app for all 13 weeks. Weekly “usefulness” ratings among the HCPS averaged 3.6 (scale: 1 “not at all useful” to 5 “very useful”). Conclusions: Study results exceeded expectations for patient acceptability and engagement with the app; as well as perceived usefulness of the portal among HCPs – suggesting the platform as an impactful tool for patients working with HCPs to manage treatment journeys. A larger study using a comparator arm will help further evaluate the impact on costs and clinical outcomes. 1. Basch E., JAMA 2017. 2. Statista, 2023.

P625 Electronic patient-reported outcomes in Inflammatory Bowel Diseases on vedolizumab therapy: Interim analysis of the non-interventional German LISTEN II study

Abstract Background Integrating systematic monitoring of patient-reported outcomes (PROs) with clinical information may enhance disease management and improve patient outcomes. Vedolizumab (VDZ) is approved for treating patients with ulcerative colitis (UC) and Crohn’s disease (CD). The German, multicentre, prospective, non-interventional LISTEN II study evaluates patient characteristics and effectiveness of VDZ treatment in real-world clinical practice, focusing on PROs. Methods Adult UC or CD patients, who either initiate VDZ intravenously (IV) or switch from IV maintenance to subcutaneous (SC) formulation, are enrolled. Data on patient characteristics, modalities of VDZ use and disease activity are recorded quarterly over one year. PROs are recorded via weekly questionnaires in app (ePRO). Sustained ePRO use was defined as proportion of patients reporting at least one PRO for ≥80% of available questionnaires. Interim data up to 9 months follow-up are presented. Results Data of 299 UC and 174 CD patients (51% and 59% female, respectively) with a mean age of 40 years were analysed. VDZ treatment was newly initiated IV (start) in 259 UC and 151 CD patients and switched from IV to SC application (switch) by 40 UC and 23 CD patients at baseline. Total mean pMayo score decreased in UC start-patients from 5.0 to 1.7 and from 1.1 to 0.8 in switch-patients. In CD start-patients HBI score decreased from 5.5 to 2.5 and from 3.1 to 2.3 in switch-patients. The ePRO was initially utilized by 226 UC and 124 CD patients (51% and 61% female, 57% and 52% sustained ePRO users, respectively) with a mean age of 39 years; 86% were start- and 14% switch-patients. Disease activity at baseline was comparable to the overall population, showing a total mean pMayo score of 4.5 for UC and a HBI of 5.3 for CD patients. Most UC (82%) and CD patients (88%) used a patient support program for the first time. ePRO use declined to approx. 40% usage within the second quarter and to 30% during further follow-up. Overall, mean symptom scores reported via the ePRO declined during the 9-month follow-up period. The most notable improvements (baseline to month 9) were reported for the symptom impact on work productivity (from 3.9 to 1.7 points) in UC patients and for sick leave (from 1.3 to 0.4 points) in CD patients (Table). For most PROs reduction in symptom scores was apparent within the first week of ePRO-use (Figure). Conclusion LISTEN II real-world data confirm VDZ effectiveness in UC and CD patients, with reduction in disease activity in IV starters and maintained effectiveness in IV-to-SC switchers. Both UC and CD patients reported sustained symptom improvement under VDZ treatment, with scores indicating improvement within the first week of ePRO-use.

Pilot trial of an electronic patient-reported outcome monitoring system in patients with metastatic breast cancer undergoing chemotherapy.

Electronic patient-reported outcomes monitoring (ePROM) is a useful communication tool for patients and healthcare providers in cancer chemotherapy. In this study, we examined the feasibility of our newly developed ePROM system, which we refer to as "Hibilog". An ePROM app was developed by extracting 18 items from the Patient-Reported Outcome-Common Terminology Criteria for Adverse Events (PRO-CTCAE). Symptom monitoring was conducted every two weeks for patients with metastatic breast cancer undergoing chemotherapy. The primary outcome was the response rate to the ePROM system. The secondary outcomes were response time, item missing rate, and distribution of responses for each symptom. A total of 71 cases (mean age 52.6years) were analyzed. Performance status was 0 in 76% of the cases and 1 or higher in 24%. First-line treatment was being administered in 30% of cases, second-line treatment in 17%, and third-line or higher treatment in 53%. The response rate to the ePROM system from registration to week 40 remained high at around 80%, indicating good compliance. The average response time was 5.5min and the missing rate for each item was below 0.4%. Among 1,093 responses, the top 3 symptoms causing interference with daily life were Fatigue (63%), Numbness and tingling (48%), and General pain (46%). Our developed ePROM system was able to capture symptoms accurately in patients with metastatic breast cancer undergoing chemotherapy while maintaining a high response compliance.

Behavioural digital biomarkers enable real-time monitoring of patient-reported outcomes: a substudy of the multicenter, prospective observational SafeHeart study.

Patient-reported outcome measures (PROMs) serve multiple purposes, including shared decision-making and patient communication, treatment monitoring and health-technology assessment. Patient monitoring using PROMs is constrained by recall and non-response bias, respondent burden and missing data. We evaluated the potential of behavioural digital biomarkers obtained from a wearable accelerometer to achieve personalised predictions of PROMs. Data from the multicenter, prospective SafeHeart study conducted at Amsterdam University Medical Center in the Netherlands and Copenhagen University Hospital, Rigshospitalet in Copenhagen, Denmark, was used. The study enrolled patients with an implantable cardioverter defibrillator (ICD) between May 2021 and September 2022 who then wore wearable devices with raw acceleration output to capture digital biomarkers reflecting physical behaviour. To collect PROMs, patients received the KCCQ and EQ5D-5L questionnaire at two instances; baseline and after 6 months. Multivariable Tobit regression models were used to explore associations between digital biomarkers and PROMs, specifically whether digital biomarkers could enable PROM prediction. The study population consisted of 303 patients (mean age 62.9±10.9 years, 81.2% male). Digital biomarkers showed significant correlations to patient-reported physical and social limitations, severity and frequency of symptoms and quality of life. Prospective validation of the Tobit models indicated moderate correlations between the observed and predicted scores for KCCQ (concordance correlation coefficient (CCC) = 0.49, mean difference: 1.07 points) and EQ5D-5L (CCC=0.38, mean difference 0.02 points). Wearable digital biomarkers correlate with PROMs, and may be leveraged for real-time prediction. These findings hold promise for monitoring of PROMs through wearable accelerometers.

Monitoring Patient Reported Outcomes in PNH: Interim Results of a Market Research with florio ® PNH

Background: Patients withparoxysmal nocturnal hemoglobinuria (PNH), an ultra-rare, acquired disorder, report various cardiovascular, gastrointestinal, neurological or hematological symptoms that can severely impact their physical functioning and health-related quality of life. Patients may require long term treatment with anti-complement therapies to alleviate PNH symptoms or complications. Whilst blood biomarkers can aid the monitoring of disease progression, patient reported outcomes (PRO) and related symptoms are also of value in assessing response to therapy, but they are not always continuously tracked by patients. Hence florio PNH, a software medical device (smartphone app), was designed to allow easy monitoring of PRO and tracking of medication by patients. It allows documentation of therapy and common symptoms as well as hemoglobin and LDH levels. florio PNH can send treatment reminders and compiles the entered data into a patient report. Patients were invited to participate in market research of florio PNH to better understand monitoring habits and expectations towards an app in this area. Methods: Twenty patients were invited to participate in market research sponsored by Florio GmbH. The participants came from the UK PNH service centre in Leeds and were selected by the coordinating clinical team at routine clinic visits. The research was submitted for ethics approval and written informed consent was provided. The market research consisted of a one hour entry interview assessing current behavior and expectations towards monitoring of outcomes, followed by an eight-week period of field-testing florio PNH app. The subsequent one hour exit interview explored the user experience with the app, and which app features were seen of value, of less value or were missing. A total of 20 patients are to be enrolled in the research, of which 17 had completed the initial interview at the time of this report. Results: The majority of participants were 50 years of age or older (59%) and described themselves as “not tech savvy”. Six participants rated the impact of PNH on their life as significant, 6 as somewhat and 5 as little to none. Fatigue was named by 10 patients unprompted as the prominent symptom affecting their social and work life. Before the research, three participants neither tracked symptoms nor reported medication, five tracked both, seven only tracked their anti-complement infusions and two only major events when they happened. Forty seven percent of participants (8 of 17) did not prepare information at all for clinical consultations in advance, as they either experienced few events or relied on their memory. All users managed to install the app, set up their account and log events without problem. The app was described as easy to use, straightforward and participants appreciated that all data was in one location and possible to summarize into a report. Sixteen (94%) patients said that they believe the app will help them track events and that they would be enthused to use it in the future. Conclusions: Monitoring applications are employed in several rare bleeding disorders and have been shown to be valuable assets for collecting patient reported outcomes, especially in the outpatient setting. Participants of this research had no experience with using disease specific apps for collecting outcomes, and usually rely on the calendar functions provided by their smartphones or paper diaries. 94% concluded that the app will help them organize their PNH data, share data with their treating clinical team, better plan their future activities and be reminded when the next treatments are due.

Mixed methods study of the feasibility and acceptability of remote monitoring of lung cancer patient-reported outcomes.

341 Background: Symptom monitoring using electronic patient-reported outcome (ePRO) systems during cancer care has been shown to provide clinical benefits and is now recommended for routine care. Integrating existing ePRO platforms into practices may lower costs and logistical barriers to use. The purpose of this study was to assess the feasibility and acceptability of a mobile app (Moovcare) for care of patients with lung cancer. Methods: In this single-site study, patients on treatment for lung cancer were recruited for 6 months of ePRO monitoring using Moovcare, an application originally designed for patients undergoing surveillance. Patients reported symptoms weekly via a survey delivered to their email address. Providers received email alerts for concerning symptoms and accessed graphical reports displaying responses over time. Providers were asked to complete an interview after 6 months of experience using Moovcare. Feasibility was assessed by the % of surveys completed per patient. Clinician acceptability was evaluated using semi-structured interviews. Results: 90 patients were recruited to participate and 46/90 (51.1%) were enrolled. 41/46 (89%) completed at least 1 survey. Patients were a mean 61.5 years of age. The majority were female (61.0%), White (73.2%), and had advanced NSCLC (92.7%). Nearly half (43.9%) had completed at least college level education. Most reported daily internet (87.8%) and email (63.4%) use. Survey completion rates are in Table 1. Alerts occurred in 78.0% of participants, with a mean of 4.2 alerts (SD=4.7) per patient. Alerts were closed by providers in a mean 2.7 days (SD=3.9). 3/7 providers participated in alert management, all of whom participated in interviews. These providers felt there was potential value for Moovcare to monitor symptoms but did find enrolled patients were already proactive about contacting the clinic for symptom management. Most interviewed providers (66.0%; 2/3) would like to use the system again and would recommend the system to others. EMR integration was strongly desired to improve workflow. Conclusions: ePRO survey completion and alert management using an existing web-based application were feasible for lung cancer patients and their providers. While concerning symptoms were commonly experienced by patients, provider perceptions of alert utility were low. Adjusting alert criteria to increase the frequency of actionable alerts and using strategies to increase ePRO use in patients with low participation levels may increase the utility of ePROs. EMR integration may improve acceptability to providers. Clinical trial information: NCT05011890 .[Table: see text]

Patient-reported symptoms and interest in symptom monitoring in HCC treated with locoregional therapies: A qualitative study.

Patient-reported outcomes (PRO) measures relevant to domains most important to patients with HCC who received locoregional therapies are needed to advance patient-centered research. Furthermore, electronic PRO monitoring in clinical care has been shown to reduce hospitalizations and deaths in patients with other cancers. We conducted a qualitative study among patients with HCC who recently received locoregional therapies to (1) identify common and distressing posttreatment symptoms to prioritize PRO domain selection and (2) gauge interest in an electronic PRO symptom monitoring system. We performed semi-structured telephone interviews among adult patients who received locoregional therapies (median of 26 days after treatment) for treatment-naïve HCC at a single tertiary care center. Interviews were conducted until thematic saturation was reached. Qualitative content analysis was conducted to identify emerging themes and sub-themes. Ten of 26 patients (38%) reported at least 1 symptom before treatment. In contrast, all participants (n = 26) with recently treated HCC reported at least 1 posttreatment physical symptom, with the most common being appetite loss (73%), fatigue (58%), abdominal pain (46%), and nausea (35%). Most participants (77%) stated they saw potential benefits in posttreatment ePRO symptom monitoring. Posttreatment symptoms after HCC locoregional therapies are common and often severe. These data can inform and prioritize PRO domain selection. Patients are interested in ePRO monitoring to monitor and proactively address posttreatment symptoms. Given the clinical benefits in patients with metastatic cancers, ePRO monitoring warrants investigation in patients with HCC.

SAT291 Remote Monitoring Of Biometric, Proteomic, And Patient-reported Outcomes In Adrenal Insufficiency

Abstract Disclosure: A. Ben-Shlomo: None. B. Chazarin: None. G. Gresham: None. M. Choudhary: None. K. Barnhill: None. K. Haptonstall: None. J. Van Eyk: None. Background: Damaged hypothalamic-pituitary adrenal axis may result in adrenal insufficiency (AI) and require hydrocortisone replacement. Currently, there are no biometric or circulating biomarkers to assess hydrocortisone replacement adequacy. Dosing is based on symptomatology, often resulting in over- or undertreatment. Methods: We used remote monitoring precision health solutions to identify AI molecular and biometric biomarkers over 2 weeks. Using remote sampling devices (Mitra Neoteryx), 10-µL dried blood samples were collected weekly for protein analysis by AI subjects before and 5 hours after daily hydrocortisone doses and age- and sex matched adrenal sufficient (AS) subjects at approximately the same times. Continuous physical activity were measured with a wearable biosensor (Fitbit Charge HR) along with remote systolic (SBP) and diastolic (DBP) blood pressure and heart rate (HR) (Omron Evolv). Self-reported AddiQoL questionnaires were completed twice daily. For biometric endpoints and questionnaires, data are summarized as mean ± SD using two-sided t-tests, where a p value &amp;lt;0.05 is considered statistically significant. For proteomic analysis, median value was calculated, and statistical testing identified differentially expressed proteins (DEPs; FDR&amp;lt;5%). Results: Fifteen AI (6 primary AI [PAI] and 9 secondary AI [SAI] and 15 AS subjects completed the study. AI group mean age was 46.3±15.7 and AS was 41.7±13.2. In both groups, 80% were female. Adherence to home monitoring was 100% in AI subjects and &amp;gt;90% in AS (13/15 assessable for biometrics (activity and BP) and 14/15 for AddiQoL). SBP was lower in PAI (106.2±7.9/68.3±7.6 [n=5]) vs SAI (119.1±15.3/71.1±8.9 [n=8]; p=0.04). Nighttime BP (22:00-05:00) for both PAI (102.3±7.8/68.5±7.9) and SAI (101.2±8.2/63.8±9) were significantly lower than AS (115.5±15.6; p=0.04 and p=0.02, respectively). AI heart rate was lower vs AS at night (61.4±11.6 [n=13] vs 72.5±11.1 [n=11]; p=0.01), especially in PAI (57.7±5 [n=5]; p=0.001). Average daily activity was higher in AS than AI as measured by steps (9010 vs 7787; p=0.02), miles (3.94 vs 3.36; p=0.03) and stair flights (14.19 vs 9.85; p=0.01). On AddiQoL, AI subjects scored lower than AS subjects (126.8±18.1 vs 143.3±14.9, respectively; p&amp;lt;0.0001). Across all dried blood samples, 444 proteins were quantified. Comparing AI vs AS, 78 were identified before morning hydrocortisone dose vs 88 before afternoon dose. Forty-nine DEPs were commonly identified and showed the same regulation trends. Nine DEPs before and 18 DEPs after morning and afternoon dosing, respectively, were identified in AI. None of these DEPs were common, indicating potential blood proteome signature changes in the morning and in the afternoon. Conclusion: Remote monitoring of biometrics and blood proteome in AI is feasible and informative and may identify biomarkers to optimize hydrocortisone replacement therapy. Presentation: Saturday, June 17, 2023

CN3 Patient adherence to digital patient-reported outcome monitoring (PROM) in cancer immunotherapies

CN3 Patient adherence to digital patient-reported outcome monitoring (PROM) in cancer immunotherapies

Electronic Patient-Reported Outcome Monitoring to Improve Quality of Life After Joint Replacement

Although remote patient-reported outcome measure (PROM) monitoring has shown promising results in cancer care, there is a lack of research on PROM monitoring in orthopedics. To determine whether PROM monitoring can improve health outcomes for patients with joint replacement compared with the standard of care. A 2-group, patient-level randomized clinical trial (PROMoting Quality) across 9 German hospitals recruited patients aged 18 years or older with primary hip or knee replacement from October 1, 2019, to December 31, 2020, with follow-up until March 31, 2022. Intervention and control groups received the standard of care and PROMs at hospital admission, discharge, and 12 months after surgery. In addition, the intervention group received PROMs at 1, 3, and 6 months after surgery. Based on prespecified PROM score thresholds, at these times, an automated alert signaled critical recovery paths to hospital study nurses. On notification, study nurses contacted patients and referred them to their physicians if necessary. The prespecified outcomes were the mean change in PROM scores (European Quality of Life 5-Dimension 5-Level version [EQ-5D-5L; range, -0.661 to 1.0, with higher values indicating higher levels of health-related quality of life (HRQOL)], European Quality of Life Visual Analogue Scale [EQ-VAS; range, 0-100, with higher values indicating higher levels of HRQOL], Hip Disability and Osteoarthritis Outcome Score-Physical Function Shortform [HOOS-PS; range, 0-100, with lower values indicating lower physical impairment] or Knee Injury and Osteoarthritis Outcome Score-Physical Function Shortform [KOOS-PS; range, 0-100, with lower values indicating lower physical impairment], Patient-Reported Outcomes Measurement Information System [PROMIS]-fatigue [range, 33.7-75.8, with lower values indicating lower levels of fatigue], and PROMIS-depression [range, 41-79.4, with lower values indicating lower levels of depression]) from baseline to 12 months after surgery. Analysis was on an intention-to-treat basis. The study included 3697 patients with hip replacement (mean [SD] age, 65.8 [10.6] years; 2065 women [55.9%]) and 3110 patients with knee replacement (mean [SD] age, 66.0 [9.2] years; 1669 women [53.7%]). Exploratory analyses showed significantly better health outcomes in the intervention group on all PROMs except the EQ-5D-5L among patients with hip replacement, with a 2.10-point increase on the EQ-VAS in the intervention group compared with the control group (HOOS-PS, -1.86 points; PROMIS-fatigue, -0.69 points; PROMIS-depression, -0.57 points). Patients in the intervention group with knee replacement had a 1.24-point increase on the EQ-VAS, as well as significantly better scores on the KOOS-PS (-0.99 points) and PROMIS-fatigue (-0.84 points) compared with the control group. Mixed-effect models showed a significant difference in improvement on the EQ-VAS (hip replacement: effect estimate [EE], 1.66 [95% CI, 0.58-2.74]; knee replacement: EE, 1.71 [95% CI, 0.53-2.90]) and PROMIS-fatigue (hip replacement: EE, -0.65 [95% CI, -1.12 to -0.18]; knee replacement: EE, -0.71 [95% CI, -1.23 to -0.20]). The PROMIS-depression score was significantly reduced in the hip replacement group (EE, -0.60 [95% CI, -1.01 to -0.18]). In this randomized clinical trial, the PROM-based monitoring intervention led to a small improvement in HRQOL and fatigue among patients with hip or knee replacement, as well as in depression among patients with hip replacement. Deutsches Register Klinischer Studien ID: DRKS00019916.

Electronic patient-reported outcome (e-PRO) monitoring for adverse event management during cabozantinib treatment in patients with advanced renal cell carcinoma: protocol for a three-arm, randomised, multicentre phase II trial (e-PRO vs paper-PRO or usual care)

IntroductionCabozantinib monotherapy is an option for treatment of advanced renal cell carcinoma (RCC). However, cabozantinib dose modification and discontinuation due to symptomatic adverse events (AEs) remains a challenge. The use...

Paper 37: Peri-Operative Complications, Safety, and Early Outcomes of a Novel ACL+ALL Reconstruction Technique using Iliotibial Band Autograft vs. Patellar Tendon ACL Reconstruction: A Retrospective Pilot Study of the SATURN (Skeletally-Mature ACLR Technique Using Reinforcement Network) Study Group

Objectives: The iliotibial band (ITB) autograft has been historically used for physeal-sparing ACL reconstructions (ACLR) in skeletally immature children (Modified-Macintosh/Micheli-Kocher technique), with an extra-articular component that replicates an anterolateral ligament reconstruction (ALLR). The procedure has demonstrated excellent return to sports (RTS), patient-reported outcomes (PROs), and relatively lower ACL graft rupture rates than other techniques in children and adolescents. However, there is only minimal evidence regarding the effectiveness of the ITB technique, or similar modifications thereof, in skeletally mature athletes. The purpose of the current study was to assess the peri-operative complications, safety, and early outcomes of a novel ITB-ACLR+ALLR technique modified for skeletally mature patients and compare it to the current gold standard of patellar tendon/bone-tendon-bone autograft technique (BTB-ACLR). The study hypothesis was that the techniques would be associated with comparably low rates of peri-operative complications, good to excellent early PROs in skeletally mature adolescents and young adults. Methods: Patients aged 13-25 years-old with closing or closed physes who underwent either primary ITB-ACLR+ALLR or primary BTB-ACLR by one of three surgeons at a single institution over a one-year period were included. The ITB-ACLR+ALLR technique utilized a 20mm wide central strip of ITB autograft, transected 100-120mm proximal to the lateral epicondyle, tubularized with whipstitches in 40mm of the free end proximally and left attached to Gerdy’s tubercle distally. The graft was passed from outside in through a complete tunnel in the lateral femoral condyle that spanned the anatomic ALL footprint extra-articularly and the anatomic ACL footprint extra-articularly, then passed through a standard tibial tunnel. The graft was affixed with outside-in cortical interference screws within both femoral and tibial tunnels. All intra-operative and post-operative complications were retrospectively recorded. Physical exam findings were collected at baseline, 3 and 6 month follow up visits, return to sport (RTS) testing was performed at 6 and 9 months, post-operatively, and patient-reported outcomes (PROs), including the Pedi-IKDC, HSS-Pedi FABS, KOOS, and EQ-VAS, were collected at baseline and 6 month follow-up visits, with ongoing collection of 12 month and 24 month follow-up PROs underway. Results: 71 patients (36 ITB, 35 BTB) were included in the current analysis. There were no significant differences in age, sex, BMI, pre-operative/baseline PROs, operative time, tourniquet time, or RTS time between the two treatment groups (Table 1). Peri-operative complications for ITB-ACLR+ALLR included 1 case of graft rupture 8 months post-operatively, which was associated with patient non-compliance with post-operative rehabilitation protocols/limitations (subsequently treated with revision BTB-ACLR), 1 case of medial meniscus tear 11 months post-operatively (subsequently treated with partial meniscectomy), 1 case of lateral thigh cramping at 6 weeks post-operatively (resolved spontaneously within 1 week), one cases of stiffness at 3 months post-operatively (resolved with dynamic bracing and PT), and one superficial wound infection at 2 weeks post-operatively (resolved with oral antibiotics). Peri-operative complications for BTB-ACLR included 4 cases of arthrofibrosis 3-5 months post-operatively (subsequently treated with arthroscopic lysis of adhesions). All 3-month Lachman and pivot-shift tests demonstrated symmetry with the contralateral knee, within 1 IKDC grade. All PROs at 6 month follow up visits were not statistically different between treatment groups (Table 2). Conclusions: Preliminary safety and clinical results suggest that a novel ACLR+ALLR technique utilizing an ITB autograft may be safe and effective for restoring knee stability and returning skeletally mature adolescents to sports, with comparable early complications and function to ACLR-BTB. Given the advantages of the ITB-ACLR+ALLR—which include technically simple graft harvest/preparation/passage/fixation, optimization of both translational and rotational stability, preservation of both the dynamic knee extensor and flexor muscle groups, and relatively smaller bony tunnels—ongoing monitoring of graft rupture rates and PROs in the current cohort and future randomized, prospective clinical trials are warranted to assess the technique in methodologically rigorous, multicenter fashion.

Patient-reported symptom monitoring linked to an alert algorithm: Effects on progression-free and overall survival in the SYMPRO-Lung trial.

12099 Background: Physician-initiated (active) monitoring of patient-reported outcomes (PROs) improves health-related quality of life (HRQOL) and overall survival (OS) in comparison to care as usual. We recently reported data from the SYMPRO-Lung trial showing for the first time that patient-initiated (reactive) PRO monitoring is equally effective as physician-initiated (active) monitoring in improving HRQOL. Here we investigate whether online PRO symptom monitoring via the active and reactive approach is also associated with improvements in 1-year progression-free survival (PFS) and OS compared to care as usual in lung cancer patients. Methods: SYMPRO-Lung is a Dutch multicenter randomized trial using a stepped wedge design. Inclusion criteria were (non-)small cell lung cancer stage I-IV, starting treatment. Patients in the intervention groups reported PRO symptoms weekly up to 1 year follow-up using the PRO-CTCAE lung cancer subset. If symptoms exceeded a pre-determined threshold, an alert was sent to a physician/nurse (active intervention group) or to the patient themselves (reactive intervention group). Survival was defined as time from start of treatment until progression (PFS), death (OS) or loss to follow-up. Follow-up time of all groups was calculated using the reverse Kaplan-Meier method. Cox proportional hazard analyses were performed to estimate the effect of PRO symptom monitoring on PFS and OS, adjusting for potential confounding (i.e. clinical characteristics and baseline EORTC QLQ-C30 domain scores). Results: 515 patients were included in the study (266 control, 249 intervention). The most pronounced uneven distribution in baseline characteristics was that the active intervention group had significantly more stage IV patients compared to the control group and the reactive intervention group (63% vs. 44% vs. 44%, p = 0.001). The median follow-up duration was 12 months for all groups. Age, sex, cancer staging, ECOG performance status and some baseline EORTC domain scores affected the association and were included as covariates in the analyses. Although not significant, we observed a tendency towards a PFS benefit for the active intervention group compared to care as usual (HR .78, 95%CI: .58 – 1.04) and an OS benefit for both of the intervention groups compared to care as usual (active: HR .80, 95% CI: .55 – 1.15; reactive: HR .69, 95% CI: .42 – 1.15). Conclusions: Although not significant, weekly PRO symptom monitoring linked to an alert algorithm showed a tendency towards a small 1-year survival benefit in lung cancer patients. Since we did not power the trial to demonstrate a survival benefit, the non-significant association might be due to a limited sample size. However, the positive results of symptom monitoring in improving HRQOL within our trial still provide sufficient evidence for implementation of PROMs in standard (lung) cancer care. Clinical trial information: NL7897 .